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2019 2
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CRISPR/Cas9-mediated excision of ALS/FTD-causing hexanucleotide repeat expansion in C9ORF72 rescues major disease mechanisms in vivo and in vitro.
Meijboom KE, Abdallah A, Fordham NP, Nagase H, Rodriguez T, Kraus C, Gendron TF, Krishnan G, Esanov R, Andrade NS, Rybin MJ, Ramic M, Stephens ZD, Edraki A, Blackwood MT, Kahriman A, Henninger N, Kocher JA, Benatar M, Brodsky MH, Petrucelli L, Gao FB, Sontheimer EJ, Brown RH, Zeier Z, Mueller C. Meijboom KE, et al. Nat Commun. 2022 Oct 21;13(1):6286. doi: 10.1038/s41467-022-33332-7. Nat Commun. 2022. PMID: 36271076 Free PMC article.
Approaches to Gene Modulation Therapy for ALS.
Meijboom KE, Brown RH. Meijboom KE, et al. Neurotherapeutics. 2022 Jul;19(4):1159-1179. doi: 10.1007/s13311-022-01285-w. Epub 2022 Sep 6. Neurotherapeutics. 2022. PMID: 36068427 Free PMC article. Review.
Poly(GR) and poly(GA) in cerebrospinal fluid as potential biomarkers for C9ORF72-ALS/FTD.
Krishnan G, Raitcheva D, Bartlett D, Prudencio M, McKenna-Yasek DM, Douthwright C, Oskarsson BE, Ladha S, King OD, Barmada SJ, Miller TM, Bowser R, Watts JK, Petrucelli L, Brown RH, Kankel MW, Gao FB. Krishnan G, et al. Nat Commun. 2022 May 19;13(1):2799. doi: 10.1038/s41467-022-30387-4. Nat Commun. 2022. PMID: 35589711 Free PMC article.
22 results